Big Phar­ma VCs jump in to back a $63M mis­sion to tack­le a ge­net­ic dri­ver of Alzheimer’s

About five years ago a pair of sci­en­tists at the Glad­stone In­sti­tutes — Robert Mahley and Yadong Huang — had seen enough clin­i­cal fail­ures in Alzheimer’s drug re­search to urge a change in strat­e­gy. If the clas­sic ap­proach on leech­ing amy­loid-be­ta out of the brain was pro­duc­ing on­ly colos­sal fail­ure, they said, why not ze­ro in on a key ge­net­ic trig­ger linked to a well-de­fined risk of de­vel­op­ing the dis­ease?

Out of that work, about three years lat­er, came a stealth biotech in South San Fran­cis­co backed by Or­bimed. A team and syn­di­cate of in­vestors came to­geth­er, in­clud­ing the ven­ture arms of some of the ma­jor play­ers in­ter­est­ed in blaz­ing a new path in the field of neu­rode­gen­er­a­tion. For two years, they’ve been qui­et­ly la­bor­ing on pre­clin­i­cal work aimed at ApoE4 — a prime cul­prit in the dis­ease — with the help of Mahley and Huang. And to­day they’re hav­ing a com­ing out par­ty of sorts, with $63 mil­lion in ven­ture cash to cel­e­brate and plans to launch the clin­i­cal work that will be need­ed to prove they may be on to some­thing.

Wel­come E-scape Bio to the Bay Area biotech com­mu­ni­ty.

The fledg­ling has some back­ers with very deep pock­ets and a pa­tient at­ti­tude about try­ing some­thing new. Along­side Or­bimed you’ll find No­vo Hold­ing A/S, John­son & John­son In­no­va­tion — JJDC, No­var­tis Ven­ture Fund and Os­age Uni­ver­si­ty Part­ners. New in­vestors in­clude Lil­ly Asia Ven­tures and Sut­ter Hill Ven­tures. In­sid­ers won’t over­look the four cor­po­rate ven­ture arms in the group.

“The con­cept was to start a new com­pa­ny fo­cused on ge­net­i­cal­ly de­fined pa­tient pop­u­la­tions in neu­rode­gen­er­a­tive dis­eases,” says Leon Chen, an Or­bimed part­ner who’s man­ag­ing the 16-mem­ber sci­en­tif­ic team. Even­tu­al­ly, Chen will step aside and go back to com­pa­ny cre­ation af­ter the back­ers find a per­ma­nent CEO.

Their re­search squad has been tin­ker­ing away on new small mol­e­cule ther­a­pies that can re­struc­ture ApoE4, linked to a much, much high­er risk of de­vel­op­ing the dis­ease, in­to a pro­tein that mim­ics ApoE3, a vari­a­tion that might well defuse a ge­net­ic dri­ver of the dis­ease.

Like a lot of start­up CEOs, Chen is leery about of­fer­ing a time­line for get­ting in­to the clin­ic with their first drug. The in­vestors have put $46 mil­lion in­to the com­pa­ny and an­oth­er $17 mil­lion tranche is wait­ing to fin­ish as­sem­bling the pre­clin­i­cal pipeline with mul­ti­ple drug can­di­dates for both Alzheimer’s and Parkin­son’s dis­ease. Then they can start hu­man test­ing in search of that first look at hu­man proof-of-con­cept da­ta.

Alzheimer’s is one of the tough­est tar­gets in bio­phar­ma. Late-stage piv­otal stud­ies have been big and mon­u­men­tal­ly ex­pen­sive. A ge­net­ic ap­proach — a no­tion that al­so helped in­spire Ryan Watts’ group at De­nali, a South San Fran­cis­co play­er op­er­at­ed by a bunch of Genen­tech vets — could be done with a small­er pa­tient pop­u­la­tion, much more nar­row­ly de­fined. In some of the ear­ly Alzheimer’s stud­ies, re­searchers didn’t have the di­ag­nos­tic tools avail­able to even make sure all the pa­tients in their stud­ies ac­tu­al­ly had the dis­ease. It was a sure fire way to wors­en the odds against suc­cess.

Chen quick­ly coun­tered my sug­ges­tion that a drug tar­get­ing the struc­ture of ApoE4 would have to be used years ahead of any symp­toms. Their work, he says, sug­gests that a drug like that could have a sig­nif­i­cant im­pact at the ear­ly stage of dis­ease de­vel­op­ment, ex­act­ly where most in­ves­ti­ga­tors are fo­cused these days, be­fore Alzheimer’s wipes out mem­o­ry.

It’s a bold plan. And they have the mon­ey to put it to a crit­i­cal test.

Novotech CRO Ex­pands Chi­na Team as Biotech De­mand for Clin­i­cal Tri­als In­creas­es up to 79%

An increase in demand of up to 79% for clinical trials in China has prompted Novotech the Asia-Pacific CRO to rapidly expand the China team, appointing expert local clinical executives to their Shanghai and Hong Kong offices. The company is planning to expand their team by 30% over the next quarter.

Novotech China has seen considerable demand recently which is borne out by research from GlobalData:
A global migration of clinical research is occurring from high-income countries to low and middle-income countries with emerging economies. Over the period 2017 to 2018, for example, the number of clinical trial sites opened by biotech companies in Asia-Pacific increased by 35% compared to 8% in the rest of the world, with growth as high as 79% in China.
Novotech CEO Dr John Moller said China offers the largest population in the world, rapid economic growth, and an increasing willingness by government to invest in research and development.
Novotech’s 23 years of experience working in the region means we are the ideal CRO partner for USA biotechs wanting to tap the research expertise and opportunities that China offers.
There are over 22,000 active investigators in Greater China, with about 5,000 investigators with experience on at least 3 studies (source GlobalData).

Daniel O'Day [via AP Images]

UP­DAT­ED: Gilead un­leash­es a $5B late-stage cash al­liance with Gala­pa­gos — lay­ing out O'­Day's R&D strat­e­gy

Daniel O’Day is executing his first major development deal since taking over as CEO of Gilead $GILD. And he’s going in deep to ally himself with a longstanding partner.

O’Day announced today that he is spending $5 billion in cash to add new late-stage drugs to Gilead’s pipeline, picking up rights to Galapagos’ $GLPG Phase III IPF drug GLPG1690 alongside adoption of the biotech’s Phase IIb drug GLPG1972 for osteoarthritis. And Gilead is also putting billions more on the table for milestones, gaining options for everything else in Galapagos’ pipeline, with a shot at all rights outside of Europe.

Altogether, Gilead is gaining rights to 6 clinical-stage assets, 20 preclinical programs and everything else being hatched in translation.

Endpoints News

Basic subscription required

Unlock this story instantly and join 54,600+ biopharma pros reading Endpoints daily — and it's free.

Hal Barron [File photo]

Hal Bar­ron's team at GSK scores a win with pos­i­tive Ze­ju­la PhI­II front­line study — now comes the hard part

Score one for Hal Barron and the new R&D team steering GlaxoSmithKline’s pipeline.

The pharma giant reported this morning that its recently acquired PARP, Zejula (niraparib), hit the primary endpoint on progression-free survival in a frontline maintenance setting for women suffering ovarian cancer — following chemo and regardless of their BRCA status.

GSK bet $5 billion on the Tesaro buyout primarily to get this drug, drawing the shaking heads of biopharma. Why pay a big premium for a drug like this when AstraZeneca was going from strength to strength with Lynparza, ran the argument, having won a hugely important accelerated approval to jump out ahead — way ahead — of the rest of the PARP players? Lynparza — now co-owned by a powerhouse cancer team at Merck — won the first approval in frontline maintenance in ovarian cancer.

Alk­er­mes adds bipo­lar I dis­or­der to its FDA wish­list; Con­go con­firms first Ebo­la case in large city

→ An ever-ambitious Alkermes $ALKS team plans to add bipolar I disorder to its list of conditions for ALKS-3831, which it plans to pitch to the FDA in Q4. Alkermes says they were persuaded to add bipolar I disorder after a pre-NDA meeting with the agency, which came about 7 months after the biotech reported positive data for schizophrenia. The drug is a combo using olanzapine/samidorphan, which they hope will be shown to be as effective as olanzapine without the substantial increase in the risk of weight gain.

Pe­ter Kolchin­sky and Raj Shah raise a $300M fund de­vot­ed to biotech star­tups

Peter Kolchinsky and Raj Shah have another $300 million-plus to play with on the biotech venture side of their investment business. 

The two announced Monday morning that they’ve put together their first pure-play venture fund at RA Capital Management, which has been known to bet on just about every angle in healthcare investing — from rounds to follow-on investments at public companies. This new fund of theirs arrives well into a go-go era of new startup financing, with a particular focus on building new biotechs.

Boehringer buys Swiss biotech in its lat­est M&A deal, go­ing the next-gen can­cer vac­cine route

Boehringer Ingelheim has snapped up a Swiss biotech startup and added their group as a new platform for the oncology pipeline. 

The German biopharma company has bagged Geneva-based AMAL Therapeutics, paying out an unspecified upfront in a $358 million deal — cash, milestones and everything else, all in. Plus there’s 100 million euros on the line for commercial milestones.

Endpoints News

Basic subscription required

Unlock this story instantly and join 54,600+ biopharma pros reading Endpoints daily — and it's free.

Ab­b­Vie beefs up the on­col­o­gy pipeline, bag­ging an up­start STING play­er with its own unique ap­proach

AbbVie isn’t letting its $63 billion buyout of Allergan stop its M&A/deals team from continuing their work.

Monday morning we learned that the pharma giant is snapping up tiny Mavupharma out of Seattle, a Frazier-backed startup that has its own unique take on STING — which is on the threshold of their first clinical trial.

Endpoints News

Basic subscription required

Unlock this story instantly and join 54,600+ biopharma pros reading Endpoints daily — and it's free.

Billing it­self as the first AI biotech to launch hu­man tri­als, Re­cur­sion adds $121M C round

Billing itself as the first AI biotech with programs in the clinic, Salt Lake City-based Recursion now has a $121 million bankroll to start gathering human data to see if it’s on the right track. 

“We’re trying to build this discovery engine,” Recursion CEO Chris Gibson tells me ahead of the C round news. “We now have the first two programs in the clinic.” And that, he adds, qualifies as a first for any AI establishment “that actually have something in the clinic.”

FDA bats back As­traZeneca's SGLT di­a­betes drug for Type 1 di­a­betes — block­ing a class on safe­ty fears

The FDA has just fired its latest salvo at the SGLT class of diabetes drugs, blowing up some commercial opportunity at AstraZeneca as part of the collateral damage.

The pharma giant reported early Monday that the FDA has rejected its blockbuster drug Farxiga for Type 1 diabetes that can’t be controlled by insulin. And while the pharma giant maintained its usual grim silence in the face of a setback, this one should be easy to interpret.